News ProQR Creates New Company, Wings Therapeutics, to Assume Development of QR-313 for Dystrophic EB

News Rare Diseases Constitute a ‘Public Health Issue,’ NCATS Director Warns

News Castle Creek, Fibrocell Teaming Up to Advance FCX-007 for Recessive Dystrophic Epidermolysis Bullosa

News PTR-01 Granted FDA’s Fast Track Status for Treatment of Recessive Dystrophic Epidermolysis Bullosa

News Approval of Off-the-Shelf Stem Cell Therapy to Treat Epidermolysis Bullosa Sought in Japan, JCR Pharmaceuticals Says

News Investigational Gene Therapy KB103 Receives Priority Designation in Europe for Dystrophic Epidermolysis Bullosa

News Third Annual ‘Believe in Brady’ Fundraiser to Benefit EB Research Partnership

News Rigosertib May Be Effective, Safe for Squamous Cell Carcinoma in RDEB Patients, Study Suggests

News RDEB Patients at Higher Risk of Osteoporosis, Fractures, Study Suggests

News JEB Involving Kidneys, Lungs Should be Classified as Mixed Form of Disease, Case Report Suggests

News First Patients Dosed in Phase 1/2 Trial Testing PTR-01 for Recessive Dystrophic Epidermolysis Bullosa

News Brexit Could Have Real Effects for UK Rare Disease Patients, Experts Warn

News Moral Dilemmas Complicate Treatment of Rare Diseases, Says Israeli Bioethicist

News NIH Rare Disease Day Highlights Joint Networks Advancing Array of Research

News Prognosis Good for Pediatric Autoimmune Blistering Diseases, but Long-Term Follow-up Needed, Study Says

News WODC 2019 Organizers Expect 1,200 to Attend Rare Disease Conference in April

News Care, Management of EB Simplex Infant During Eye Surgery Detailed in Case Report

News Vienna to Host RARE2019 Meeting on Rare Diseases

News Mechanism Underlying Fibrosis in RDEB Could Be New Therapeutic Target

News Blocking Inflammatory Pathway Reduces Blisters in EB Simplex Generalized Severe Patients

News Patients, Family and Friends to Take Part in Variety of Rare Disease Day Events Worldwide

News Steroid-induced Diabetes in EBA Patient Shows Importance of Follow-up Care

News Cannabinoids Could Help Manage EB-related Pain, Study Suggests

News Next-generation DNA Sequencing Efficiently Identifies Novel Mutation Causing Junctional EB in Newborn

News Clinical Diagnosis More Accurate Than Skin Biopsy in Inherited EB Patients, Study Reports

News Severe EB Requires Multidisciplinary Approach, Case Report Suggests

News Decorin Extends Survival, Eases Skin Fibrosis in Mouse Model of RDEB

News Krystal Biotech Completes New Plant Construction to Support KB103 Development in Treating DEB

News Independent Committee Supports Additional Patient Enrollment in Phase 3 Trial of AP101 in EB Patients

News Researchers Create Dressing Glove for EB Based on Patients’ Feedback

News Diagnostic Testing More Common in Most Severe EB Types, Study Reports

News Collagen XVII Mutation Behind Skin Blistering Seen in Mild JEB, Study Confirms

News Four Patients Treated in Phase 2 Trial Testing Topical Gene Therapy, KB103, for Wound Healing

News Rising Healthcare Costs Strain EU Budgets Even as New Therapies Flourish

News EB Groups Give Fibrocell $900,000 to Advance Gene Therapy in Clinical Trials

News Phase 3 Trial of RDEB Therapy EB-101 to Start in 2019, Abeona Therapeutics Says

News TXA127 Enters Development Program as Potential Treatment for EB

News Diacerein Ointment Could Be Safe Treatment for Generalized Severe EBS, Study Suggests

News Intravenous Immunoglobulin and Rituximab Most Effective EBA Treatments, Study Reports

News RegeneRX, GtreeBNT Provide Update on RGN-137 for Treatment of Epidermolysis Bullosa

News #NORDSummit – Major Issues on Table for Rare Disease Patients in US as Midterm Elections Approach

News FDA Advises Fibrocell on Phase 3 Trial Design for FCX-007, Potential RDEB Therapy

News TWi Biotechnology Enrolls First Patient in Phase 2 Trial Testing AC-203 for EB Treatment

News DNA Sequencing Technique Identifies Two New Mutations in Patient with Simplex and Junctional EB

News First RDEB Adult Patients Do Well in Clinical Trial with Gene Therapy KB103

News Advocacy Groups, Doctors Question Rising Prices of Rare Disease Treatments

News Gene Editing Corrects Mutation Causing Recessive Dystrophic Epidermolysis Bullosa, Study Reports

News Calcipotriol Ointment Seen to Improve Wound Healing in RDEB Patient, Study Finds

News Fibrocell Awarded $1.4M from FDA to Advance FCX-007 in Clinical Trials for RDEB

News #NORDSummit – More Than 700 Expected to Attend Oct. 15-16 Rare Disease Summit in Washington

News Dad Who Lost Baby to EB Nears Final Stretch in ‘Big Ride’ to Raise Money to Combat Disease

News Specific Enzymes Cause Mutations That Fuel Aggressive Skin Cancer in Patients With RDEB, Study Shows

News Amryt’s AP101 for Treating EB Receives IND Clearance from FDA

News RDEB Is Main Suspect Behind SCC, a Rare Form of Skin Cancer, Study Shows

News 2 EB Research Groups Based Continents Apart Partner to Advance New Treatments

News Case Report Describes Successful Treatment of JEB with Terrasil Ointment

News Austrian Epidermolysis Bullosa Patient Inspires Dad to Establish World’s First ‘EB House’

News FDA Grants Fast Track Status to Castle Creek’s CCP-020 for Treatment of EBS

News Joint Venture Plans to Advance Potential EB Therapy RGN-137 into Phase 3 Trial

News Amryt’s AP101 Granted Rare Pediatric Disease Designation for EB

News Blocking Inflammation-linked PI3K-Delta Molecule Could Lead to EBA Therapy, Mouse Study Suggests

News Antibiotic Gentamicin May Benefit About 80% of Herlitz JEB Patients, Study Says

News Cord Blood Platelet Gel Holds Potential to Treat Epidermolysis Bullosa, Study Shows

News Debra’s Family-oriented EB Care Conference Kicks Off Sunday in Arizona

News Higher Levels of Granzyme B Play Key Role in EBA, Other Skin Blistering Diseases, Study Suggests

News Case Report Highlights Rare EBA Diagnosis in 21-Year-Old Patient

News Phase 1/2 Trial of DEB Therapy Candidate QR-313 Now Enrolling Patients, ProQR Announces

News Castle Creek Enrolls First Patient in Phase 1 Trial for CCP-020 as Treatment for EBS

News 2 New Mutations Found in Family with Epidermolysis Bullosa Pruriginosa, Case Study Reports

News Allogeneic Fibroblast Injections into Skin Lesions Are Safe, Effective for RDEB, Study Shows

News ProQR to Receive Up to $5 Million from Nonprofits to Develop QR-313 for DEB

Just Published, News Serlopitant’s Potential to Manage Itch in EB Patients Shown in Exploratory Trial

News Abeona Opens Manufacturing Plant to Support EB-101, and Other Gene and Cell Therapy Candidates

News US ‘Right to Try’ Law Meets with Mix of Praise and Criticism, Including Among Those with Rare Diseases

News EB Treatment Candidate BPM 31510 Granted Orphan Drug Designation in US

News #ECRD2018 – Genome Editing Might Be ‘Cure’ for Rare Diseases But Ethical Guidelines Needed, Panel Says

News #ERDC2018 – When Treating Rare Disease Patients, Don’t Overlook Quality of Life, Panel Urges

News FDA Grants Fast-Track Designation for Krystal Biotech’s Gene Therapy Candidate KB103 to Treat DEB

News Positive Interim Results of Phase 1/2 Trial Support FCX-007 Gene Therapy for RDEB

News Trial Shows Abeona’s Gene Therapy EB-101 Helps Wounds Heal in RDEB Patients

News Diacerein Ointment Granted Rare Pediatric Disease Status for EB Treatment

News #ECRD2018 – EU Must Do More for Rare Disease Patients, Eurordis Leaders Say

News ‘Rare Impact Awards’ Dinner Marks Orphan Drug Act and NORD at 35

News Rare Case of Genetic and Autoimmune Subtypes of EB Described in Report

News Krystal Biotech Doses First Patient in Phase 1/2 Trial Testing Gene Therapy Candidate KB103 for DEB

News Collagen Vesicles Released by Bone Marrow Stem Cells May Explain Their Therapeutic Potential, Study Shows

News Epidermolysis Bullosa Can Contribute to Esophageal Stenosis, Case Study Finds

News #ECRD2018 – Patient Access and Collaboration Focus of Eurordis Meeting May 10-12

News FDA Green-lights Krystal’s KB103 Gene Therapy Trial for DEB Patients

News Human Genome Project Team at NIH Honored for Advancing Research into Genetic Diseases

News Krystal Biotech’s KB103 Receives Orphan Medicinal Product Designation in Europe

News $3.8M Defense Grant Awarded to Develop Stem Cell Therapies for EB, Wound Care

News NIH Agency Pioneers Collaborative Research into Rare Diseases

News Researchers Identify a Blood Biomarker for Epidermolysis Bullosa-associated Squamous Cell Carcinoma

News Scientists Reduce Blistering in EBA Mice by Honing In on the Immune System

News EBA Can Lead to Acute Renal Failure, Case Report Contends

News SYK Protein a Key Regulator of Inflammatory Response in EBA Mouse Model, Study Finds

News Krystal Files Application to Start Clinical Trial of Gene Therapy Candidate KB103

News Fibroblast Skin Injections Show Promise in Healing Wounds in Dystrophic Epidermolysis Bullosa Patients, Study Finds

News Amryt Pharma Advances Lead Candidate AP101 to Global Phase 3 Trial for EB